This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Epoetin Alfa in Treating Anemia in Patients Undergoing Chemotherapy for Multiple Myeloma (NCT00400686), a na na study. The primary outcome is Change From Baseline in Hemoglobin at Day 28.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00400686 |
| Phase | NA |
| Condition | Anemia, Multiple Myeloma, Plasma Cell Neoplasm |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 31 |
| Arms in the primary outcome | n/a |
| Lead sponsor | The Cleveland Clinic |
| Results posted | Yes |
2. Clinical Question
The study asked whether high-dose epoetin alfa treatment was associated with a change in hemoglobin over the first 28 days in patients with multiple myeloma and anemia who were undergoing chemotherapy. The registry record describes a single-institution, open-label, nonrandomized pilot study. It lists 31 enrolled participants and one epoetin alfa group.
Population
Patients with multiple myeloma and anemia undergoing chemotherapy. The record also lists plasma cell neoplasm among the conditions. Enrollment was 31.
Intervention
Epoetin alfa, administered as 80,000 units subcutaneously each week beginning on Day 1. The record says the dose was adjusted on Day 28 based on participants’ hemoglobin levels.
Comparator
No separate comparator group is described. The record identifies a single-group study, so it does not provide a like-for-like control comparison.
Primary question
What was the median change from baseline in hemoglobin at Day 28? The endpoint is defined, but its measured value is not reported in the registry record.
The official title also names blood transfusion requirements and quality of life as topics of interest. The identities, definitions, and results of any additional outcome measures are not reported in the registry record. Accordingly, the clinical question that can be evaluated from the posted results is limited to the study’s stated hemoglobin endpoint and the reported adverse-event counts; neither establishes a comparison with an untreated or alternative-treatment group.
3. Trial Design
The registry describes a single-institution, open-label, nonrandomized pilot study. Its model is single group, with 31 participants enrolled and one intervention listed: epoetin alfa. The study status is completed. The record lists a start of 2003-09 and a primary completion of 2009-01.
The treatment description specifies 80,000 units of epoetin alfa by subcutaneous administration once each week, beginning on Day 1. It also says the dose was adjusted on Day 28 according to participants’ hemoglobin levels. The record does not report the adjustment rules, the duration of treatment after Day 28, or further details about treatment adherence.
Because the study has no separate control group and was not randomized, changes during treatment cannot be separated from other changes over time using a concurrent comparison. Open-label means the study is described as unmasked; the record lists masking as none. How awareness of treatment may have affected measurement or reporting is not reported in the registry record.
The study is identified as a pilot and its phase is listed as NA. A sample-size justification, recruitment details, eligibility criteria, and the protocol’s statistical plan are not reported in the registry record. The title indicates a single institution, but the record does not describe the institution’s recruitment procedures or the characteristics of the enrolled participants. The registry lists three outcome measures, but only the primary hemoglobin outcome is identified in the registry entries. The names and results of other measures are not reported in the registry record.
The analysis record lists zero analyses. Thus, the record describes the study design and intended primary endpoint but does not provide an analysis result for that endpoint.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Epoetin Alfa - 80,000 U sc |
|---|---|
| STARTED | 31 |
| COMPLETED | 31 |
| NOT COMPLETED | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Epoetin Alfa - 80,000 U sc |
|---|---|---|
| Participants analysed | 31 | |
| Age, Continuous | years | 61.9 (41 to 80) |
| Sex: Female, Male: Female | Participants | 12 |
| Sex: Female, Male: Male | Participants | 19 |
| Region of Enrollment: United States | participants | 31 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Hemoglobin at Day 28, measured over Baseline to Day 28.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | g/dL |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the change from baseline in hemoglobin at Day 28, measured in g/dL. The registry says the reported summary is the median and that dispersion is described using the full range. The endpoint description frames the measurement as change after treatment with high-dose epoetin alfa. The actual median change and full range are not reported in the registry record.
A change score expresses how a measurement at follow-up differs from that participant’s baseline measurement. In general, examining change can describe movement over time while accounting for each participant’s starting value. A median summarizes the middle of the observed values after they are ordered. It can be useful when a distribution is skewed or includes extreme observations, because the median is less affected by the magnitude of extremes than the mean. The full range gives the lowest and highest observed values, but it does not show how values are distributed between those endpoints.
Hemoglobin is the specific measurement named for the primary endpoint; the trial title and rationale concern anemia. The registry does not report the endpoint’s analysis population, the number of participants with a Day 28 measurement, or a confidence interval around the median. It also does not report a threshold for interpreting a change as clinically important. The endpoint therefore identifies what the study intended to describe, but the posted record does not supply the result or its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record establishes that the primary outcome was planned as median change from baseline in hemoglobin between baseline and Day 28. It does not report the median, the full range, a confidence interval, a test statistic, or a p-value for that outcome. Consequently, the posted results do not establish the direction or magnitude of the observed hemoglobin change, or how precisely that change was estimated.
The study enrolled 31 participants in a single epoetin alfa group. That enrollment count is not necessarily the number contributing a Day 28 measurement: the number analyzed for the primary endpoint is not reported in the registry record. The registry lists three outcome measures, but the identities and findings of the other measures are not reported in the registry record. The official title mentions transfusion requirements and quality of life, but that does not by itself establish that either was analyzed or what the results were.
As a single-group, nonrandomized study, the design can describe outcomes among participants receiving the intervention but does not provide a concurrent control group for estimating what would have happened without the intervention or under another treatment. General statistical interpretation: a before-and-after change can coincide with treatment and still reflect other influences, including the course of illness, chemotherapy, or changes in care. The registry record does not provide information that would quantify or rule out such influences.
The adverse-event record lists 0 serious adverse events among 31 participants at risk and 3 other adverse events among 31 participants at risk. These are counts recorded for this group, not estimates from a comparative safety analysis. The record does not list serious adverse-event terms. Deaths are not reported in the registry record. The event counts therefore describe what was recorded, but they do not by themselves establish a comparative risk or a complete safety profile.
Overall, the registry record gives design details, a primary endpoint definition, and some adverse-event counts, but no efficacy analysis. Any interpretation of the hemoglobin endpoint must remain conditional on results that are not reported in the registry record. The study’s single-group design also limits causal interpretation independently of whether an outcome value is available.
9. Statistical Methods Explained
The registry identifies a median change in hemoglobin from baseline to Day 28 as the primary outcome, with the full range as the dispersion summary. It does not report the numerical result or a statistical analysis of that endpoint. The following concepts explain how such summaries are generally read; they do not describe an analysis documented for this study unless the registry explicitly says so.
What a median change describes
For each participant with the required measurements, a change score is based on the follow-up value relative to baseline. The median of those changes is the central value after ordering the participant-level changes. It is not the same as a change calculated from group-level medians, and it does not reveal the individual pattern of responses. The registry does not say how missing measurements, if any, were handled or confirm the number of participants contributing to the endpoint.
The full range is the span from the minimum to the maximum observed change. It can show whether observed values varied widely, but it is sensitive to extreme observations and does not indicate how many participants had values near the endpoints. A range alone is not a confidence interval. The record does not report a confidence interval, standard error, or other uncertainty estimate for the primary result.
What uncertainty measures would add
General statistical explanation: a confidence interval is an interval estimate intended to express uncertainty about a population quantity under stated assumptions. Its width reflects both the observed data and the method used to calculate it. A confidence interval for a median change would help readers assess the precision of the estimated central change; it would not resolve the lack of a control group. No confidence interval or method for estimating uncertainty is reported in the registry record.
A hypothesis test would address a prespecified statistical question under a model, such as whether an effect is compatible with a null value. A p-value, if reported, would not state the probability that the null hypothesis is true or measure the clinical importance of an effect. The registry lists zero analyses and does not report a p-value or testing procedure for the primary endpoint. It is therefore not possible to describe a trial-specific test or infer a statistical conclusion from the endpoint definition alone.
Why the design matters
In general, random allocation to concurrent groups can help balance measured and unmeasured factors across groups, while a single-group before-and-after study lacks that direct comparison. This trial is explicitly described as nonrandomized and single group. Its observations could describe what happened among enrolled participants during the study period, but the design does not isolate treatment effects from other time-related influences. The registry does not report an adjustment strategy intended to address those influences.
Open-label describes a study in which treatment masking is absent. In general, awareness of treatment can affect behavior, co-interventions, reporting, and some outcome assessments. Hemoglobin is a laboratory measure, but the record does not say whether assessment procedures were blinded or whether other safeguards were used. No claim about actual bias can be made from the masking designation alone.
Planning illustrations are not observed power
The statistical planning illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These are planning assumptions, not a description of the study’s actual single-group allocation or a report of the trial’s design rationale. The record does not report the trial’s own sample-size justification.
General statistical explanation: a planning sample size estimates how many participants a hypothetical design might require under chosen assumptions about an effect, variability, allocation, and error rates. It is not an observed result and is not observed power. A sample size calculated after observing an effect should not be interpreted as evidence that the completed study had a particular probability of detecting an effect. The primary endpoint’s observed value is not reported in the registry record, so readers cannot assess the effect assumption from that record. The two-arm equal-allocation assumption in the planning illustration also does not match the registry’s single-group study structure.
The sample-size assumptions refer to participants per arm for outcomes other than time-to-event outcomes, and total events for time-to-event outcomes. The primary endpoint is a Day 28 hemoglobin change, not a time-to-event endpoint. No specific planning sample-size result or trial-specific calculation is reported in the registry record from the registry record. Over-dispersion, which means variability greater than a model assumes, can make standard errors too small and uncertainty appear too narrow; the record does not report a model or dispersion assessment for this endpoint.
In short, the endpoint definition and design allow a reader to understand the intended measurement and important limits on interpretation. They do not supply the observed treatment-associated change, its uncertainty, or a formal test result.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists three outcome measures, but identifies only the primary endpoint in the available record. The other outcome names, their timing, and their results are not reported in the registry record. The official title refers to transfusion requirements and quality of life, but the record does not establish their status as additional analyzed outcomes or describe their measures.
Multiplicity arises when several outcomes, time points, subgroups, or statistical tests are assessed. General statistical explanation: as the number of tests increases, the chance of at least one apparently unusual result can increase if no adjustment or prespecified hierarchy is used. The record does not report a multiplicity strategy, an outcome hierarchy beyond identifying the primary endpoint, or a list of planned comparisons. It is therefore not possible to determine whether any adjustment was planned or applied.
Missing-data methods and the analysis population are not reported in the registry record. The total enrollment was 31, but the number with baseline and Day 28 hemoglobin measurements is not reported. The record does not say whether participants who discontinued, lacked measurements, or otherwise did not contribute to an endpoint were included in any analysis. It also does not describe imputation, sensitivity analyses, or rules for excluding observations.
These omissions matter because a reported change among participants with complete measurements could differ from the change among all enrolled participants if availability of measurements is related to outcomes. That is a general concern, not evidence that missing data occurred in this study. The registry lists zero analyses, so it provides no analysis-population description or missing-data assessment to evaluate.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Epoetin Alfa - 80,000 U sc | 0 | 31 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Epoetin Alfa - 80,000 U sc | 3 of 31 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Epoetin Alfa - 80,000 U sc |
|---|---|---|
| Venous Thromboembolic Event (VTE) | Blood and lymphatic system disorders | 3 of 31 |
The adverse-event record lists 31 participants at risk for serious adverse events and 0 affected. For other adverse events, it lists 31 at risk and 3 affected. No serious adverse-event terms are listed. Deaths are not reported in the registry record.
These figures describe recorded counts in the epoetin alfa group. They are not a comparison with a control group, and the record does not provide event definitions, observation duration, event-level details, or a statistical comparison. The distinction between the serious-event and other-event categories should be retained: the posted counts do not say that no adverse events of any kind occurred.
General statistical explanation: a zero count in a study does not establish that the underlying event risk is zero. With a limited number of participants, uncommon events may not occur during observation even if they can occur in a broader population. A count also does not convey timing, duration, severity within a category, or whether an event was considered related to treatment. Those details are not reported in the registry record. The counts can be read as descriptive safety reporting for this study group, not as proof of comparative safety or a complete account of harms.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed, open-label, nonrandomized, single-group pilot study of epoetin alfa in patients undergoing chemotherapy for multiple myeloma. It specifies weekly administration beginning on Day 1, with a dose adjustment on Day 28 based on hemoglobin levels. The primary endpoint is median change in hemoglobin from baseline to Day 28, with the full range intended as the dispersion summary.
The record does not report the primary endpoint value, its range, a confidence interval, or a statistical test. It therefore does not allow a reader to determine the observed magnitude or precision of hemoglobin change from the registry record. Nor does it report results for the other outcome measures. The study title names transfusion requirements and quality of life, but their outcome definitions and results are not reported in the registry record.
The design can describe outcomes observed in the treated group, but it cannot provide a direct comparison with a concurrent untreated group or another treatment. General statistical explanation: an observed change over time does not alone identify the cause of that change. Without a control group, other changes during the same period cannot be separated from an intervention-associated change using a randomized comparison.
The adverse-event entries record 0 serious adverse events among 31 at risk and 3 other adverse events among 31 at risk. These figures describe recorded events for the study group and do not establish comparative safety. The death count is not reported in the registry record.
Thus, the record supports statements about the study’s structure, planned primary measurement, and listed event counts. It does not support a numerical conclusion about the primary hemoglobin outcome, a treatment-effect estimate against a comparator, or a comprehensive account of efficacy and safety.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the difference between a clearly named endpoint and an available endpoint result. The registry specifies median change from baseline in hemoglobin at Day 28 and identifies the intended summary of dispersion, yet does not report the observed median or range. Readers can identify what was meant to be measured without being able to assess the result’s magnitude or precision.
Its single-group, nonrandomized design also illustrates the limits of interpreting change over time. A within-group change can describe participants’ measurements during treatment, but without a concurrent comparison it does not isolate the effect of treatment from other influences. This distinction remains important even when a study reports an outcome clearly.
The record’s sample-size assumptions make a separate statistical point. The planning illustration uses equal allocation between two arms and an effect defined as a fraction of an observed effect, while the registered study is single group. Such assumptions describe hypothetical planning, not the actual study design or a study-specific sample-size rationale. Planning illustrations are not observed power, and an unreported endpoint result prevents readers from evaluating the stated effect basis from the registry record.
Finally, the safety entries show why event counts need context. Counts of serious and other adverse events are descriptive, while the absence of a comparator, event details, and reported follow-up duration prevents a comparative interpretation. Together, these features make the record useful for understanding endpoint definitions, study structure, and reporting limitations, while leaving the substantive outcome questions unresolved.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
Several limitations specific to this trial follow directly from its design and the registry record. It is a single-group, nonrandomized study, with no like-for-like control comparison. Any observed before-and-after change would therefore be difficult to distinguish from other changes occurring during the same period. The open-label designation means masking was absent; details about outcome-assessment procedures are not reported in the registry record.
The primary endpoint is defined, but its result is not reported. The record does not give the median change, full range, confidence interval, analysis method, or number of participants contributing to the Day 28 result. Although enrollment was 31, the endpoint analysis population is not reported. This prevents assessment of both the observed outcome and its precision.
The record lists three outcome measures but does not identify the additional measures or report their results. A multiplicity plan, missing-data methods, subgroup analyses, interim analyses, and analysis populations are not reported in the registry record. A protocol-specific sample-size justification is also not reported; planning assumptions are not a substitute for one.
The safety record gives counts but does not provide serious-event terms, event details, observation duration, or a comparative analysis. Deaths are not reported in the registry record. Eligibility criteria, participant characteristics, and details of the Day 28 dose-adjustment rules are also not reported. These omissions limit assessment of who was studied, how treatment was adapted, and how complete the recorded outcomes may be.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Epoetin Alfa in Treating Anemia in Patients Undergoing Chemotherapy for Multiple Myeloma — NCT00400686.
- Registry record last updated 2018-02-22; status last verified 2018-01.
- Results first posted 2016-12-09.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT00400686 is a completed, single-institution, open-label, nonrandomized pilot study with 31 enrolled participants. It evaluated epoetin alfa in patients with anemia undergoing chemotherapy for multiple myeloma. The intervention description specifies 80,000 units subcutaneously each week beginning on Day 1, with dose adjustment on Day 28 based on hemoglobin levels.
The primary endpoint was median change from baseline in hemoglobin at Day 28, with full range as the dispersion measure. The endpoint result and analysis are not reported in the registry record. The record lists 0 serious adverse events among 31 at risk and 3 other adverse events among 31 at risk. Deaths are not reported in the registry record. With no separate comparator and no reported primary outcome result, the registry record does not establish a comparative treatment effect.